Long-term Follow Up of Patients Treated with D-Penicillamine in the Neonatal Period

Background: In neonatal therapy D-penicillamine (D-PA) was first recognized to have a potential benefit for neonatal hyperbilirubinemia in the early 1970s. Controlled, randomized clinical trials confirmed the effectiveness of D-PA in the treatment of haemolytic disease of the newborn and retinopathy of prematurity. The aim of this study was to explore potential long-term effects of D-PA by measuring health state of adults treated with this drug in the newborn period. Methods: The self-perceived health and health related quality of life (HRQoL) have been investigated in a cohort of 23-36 years old patients using the EuroQol5D instrument. Self-administered questionnaires were mailed comprising EQ-5D instrument and questions on academic achievements, and presence of neurosensory impairments. The original cohort consisted of 1492 subjects. 518 participants returned the questionnaires, of which 32 had to be excluded due to incomplete responses. As reference, the data of the quality of life survey was used; this study was conducted in the early years of 2000, on a representative sample of 5503 members of a Hungarian population with average achievement [22]. Results: The occurrence of neurosensory disabilities and educational levels on HRQoL were also evaluated. The authors have examined the bias between those who returned complete questionnaires and those who returned incomplete questionnaires. In all age groups of the cohort the mean Visual Analogue Scale (VAS) score was remarkable higher, whereas the mean EQ-5D index was lower than the Hungarian age specific value, which discordance can be explained by the "disability paradox". Significantly more respondents with VLBW compared to their term peers reported to have neurosensory impairment and lower educational level. Conclusions: The different characteristics of incomplete respondents can lead to large biases and through that way have great influence on HRQoL estimates. Focusing on the potential effects of D-PA, the following facts are verifiable: (i) adults survivors of prematurity can suffer from numerous pathological conditions. Consequently, their health/behaviors were significantly weaker (as it was expected) in comparison with the average population's examined [22] (ii) on the other hand, those adults who were born at term, their health/behaviors were better. This was, however, statistically not significant.